So, regarding efficacy testing, I think the costs/benefits have to be assessed in full context. If you go back to the time before the FDA, it was a time of incredible wonder drugs and useless patent medicines. Kind of like the Internet: the price of being able to put up a domain name in 10 minutes with no centralized check for accuracy means information proliferates and the web/market/search sorts it out.
And we kind of know what a safe-but-not-necessarily-effective market for drugs will look like: the supplement industry. Supplements are cheap, they vary in effectiveness on a per person basis, and they have undoubtedly produced some really great things (creatine, omega 3). Take a look at this awesome graphic:
The thing is, with centralized regulation for efficacy two things happen. First, many of the bubbles on that graph never appear in the first place. Second, because they never appear, they never accumulate enough evidence/market size to rise up the list. We are choking the channel if centralized regulators require our minimum viable products to be not just safe, but highly efficacious.
The best way to see this is that centralized regulation kills iteration. Talk to anyone in the drug space: they'd love to be able to change their dosing methodology (altering dosage amount, frequency, formulation) or otherwise take advantage of serendipitous post-market findings. Viagra, famously, was initially intended to medicate blood pressure[1].
But right now they can't even change the labels on their drugs without the FDA's approval, which is why the average layman gets a folded-up chemistry textbook[2] rather than a user-friendly instruction manual, let alone a website which totes up other people's experiences with the drug. To get a sense of how much that could contribute to the patient user experience, see Help Remedies[3], which can get away with better UI/UX because they're dealing in generics.
Anyway, on net, I think something like a pharmacogenomic erowid.org [4,5] is the best way to establish efficacy. That would be distributed and the data would be public and constantly updated, with sample sizes far in excess of the current FDA process. Patients would get accounts and link their genomic information with the site after buying any new drug, and input their own survey data in order to see other people's (aggregated, anonymized) experiences. This would mean that you can launch safe drugs of unproven efficacy, and then collect efficacy data at a far larger scale than we do today. But this kind of innovation will only be possible in a jurisdiction outside the FDA's thumb.
To take the internet domain registration metaphor further, it also requires a centralized value authority (google) in order to be navigable. In some sense, Google's primary task is spam filtering---analogous to efficacy guarantees---which enable efficient information gathering.
I don't argue that the FDA is an efficient structure for doing efficacy testing, I just think punting the value discovery/marketing process to vague distributed processes isn't a good answer.
I think the supplement market is a great example as well. Many low value treatments saturate the market and the responsibility for making decisions is democratized and difficult. Canonical sources of efficacy information might not be needed as barriers to entry, but reputation, trust, and canonization are valuable heuristics in decision making processes and this leads to power.
And we kind of know what a safe-but-not-necessarily-effective market for drugs will look like: the supplement industry. Supplements are cheap, they vary in effectiveness on a per person basis, and they have undoubtedly produced some really great things (creatine, omega 3). Take a look at this awesome graphic:
http://www.informationisbeautiful.net/play/snake-oil-supplem...
The thing is, with centralized regulation for efficacy two things happen. First, many of the bubbles on that graph never appear in the first place. Second, because they never appear, they never accumulate enough evidence/market size to rise up the list. We are choking the channel if centralized regulators require our minimum viable products to be not just safe, but highly efficacious.
The best way to see this is that centralized regulation kills iteration. Talk to anyone in the drug space: they'd love to be able to change their dosing methodology (altering dosage amount, frequency, formulation) or otherwise take advantage of serendipitous post-market findings. Viagra, famously, was initially intended to medicate blood pressure[1].
But right now they can't even change the labels on their drugs without the FDA's approval, which is why the average layman gets a folded-up chemistry textbook[2] rather than a user-friendly instruction manual, let alone a website which totes up other people's experiences with the drug. To get a sense of how much that could contribute to the patient user experience, see Help Remedies[3], which can get away with better UI/UX because they're dealing in generics.
Anyway, on net, I think something like a pharmacogenomic erowid.org [4,5] is the best way to establish efficacy. That would be distributed and the data would be public and constantly updated, with sample sizes far in excess of the current FDA process. Patients would get accounts and link their genomic information with the site after buying any new drug, and input their own survey data in order to see other people's (aggregated, anonymized) experiences. This would mean that you can launch safe drugs of unproven efficacy, and then collect efficacy data at a far larger scale than we do today. But this kind of innovation will only be possible in a jurisdiction outside the FDA's thumb.
[1] http://www.mc.vanderbilt.edu/lens/article/?id=116
[2] http://dailymed.nlm.nih.gov/
[3] http://www.helpineedhelp.com
[4] http://www.pharmgkb.org/
[5] http://www.erowid.org/